Open AccessReplication of randomized clinical trial results using real-world data: paving the way for effectiveness decisions
Author(s) -
Kristin M. Sheffield,
Nancy A Dreyer,
James F. Murray,
Douglas E. Faries,
M.N. Klopchin
Publication year - 2020
Publication title -
journal of comparative effectiveness research
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.567
H-Index - 23
eISSN - 2042-6313
pISSN - 2042-6305
DOI - 10.2217/cer-2020-0161
Subject(s) - randomized controlled trial , medicine , replicate , replication (statistics) , real world evidence , real world data , comparative effectiveness research , clinical trial , health care , medical physics , alternative medicine , risk analysis (engineering) , data science , computer science , surgery , statistics , mathematics , pathology , virology , economics , economic growth
The FDA is preparing guidance about using real-world evidence (RWE) to support decisions about product effectiveness. Several ongoing efforts aim to replicate randomized clinical trial (RCT) results using RWE with the intent of identifying circumstances and methods that provide valid evidence of drug effects. Lack of agreement may not be due to faulty methods but rather to the challenges with emulating RCTs, differences in healthcare settings and patient populations, differences in effect measures and data analysis, bias, and/or the efficacy–effectiveness gap. In fact, for some decisions, RWE may lead to better understanding of how treatments work in usual care settings than a more constrained view from RCTs. Efforts to reconcile the role and opportunities for generating complementary evidence from RWE and RCTs will advance regulatory science.